A compilation of the most pertinent information regarding the MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme. supply issue. The IPA has done its best to gather

The AMDA is writing to our Pompe patient community to let everyone know that the AMDA has been working closely

The European Medicines Agency’s (EMEA) Committee for Medicinal Products (CHMP) has recommended that infants, children and adolescents be given priority

Since the first market approval of Myozyme, the IPA has liaised directly with Genzyme LSDA group of diseases in which substances build up inside lysosomes because the body cannot break them down properly. Therapeutics to insist that an

Since the first market approval of Myozyme, the IPA has liaised directly with Genzyme LSD Therapeutics to insist that an

This guidance is being distributed to all treating physicians worldwide except those in the European Union (EU). Separate guidance will

We would like to take this opportunity to provide an important update to the Pompe Community. Background In the early

We would like to take this opportunity to provide an important update to the global Pompe Community. Background In the

The US Food and Drug Administration (FDA) has informed Genzyme that the FDA plans an accelerated approval for alglucosidase alfa

The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October